Sarepta Therapeutics Introduces Online Resource Center on Exon Skipping for the Duchenne Muscular Dystrophy Community

Sarepta Therapeutics Introduces Online Resource Center on Exon Skipping for the 
Duchenne Muscular Dystrophy Community 
CAMBRIDGE, MA -- (Marketwired) -- 10/02/13 --  Sarepta Therapeutics,
Inc. (NASDAQ: SRPT), a developer of innovative RNA-based
therapeutics, today announced a new online resource center, called
Let's Skip Ahead, for families affected by Duchenne muscular
dystrophy (DMD) and their healthcare providers. The new website,
available at, includes information and educational
resources intended to make the science of exon skipping simple, and
provides an opportunity to sign up for updates about upcoming Sarepta
clinical trials. 
"Sarepta is deeply committed to pursuing exon-skipping treatments for
patients with DMD who can potentially benefit from our technology,
and this website will help families and physicians stay informed
about our efforts," said Chris Garabedian, president and chief
executive officer of Sarepta Therapeutics. "We recognize that many in
the Duchenne community are seeking to understand exon skipping and
the promise this innovative and complex technology may hold for them.
Resources on the Let's Skip Ahead website include information tools
that can support discussions between patients, their families and the
physicians who care for them." 
Let's Skip Ahead ( also offers an exon mapping tool
that allows visitors to explore the potential link between specific
genetic mutations in DMD and exon skipping. In addition, the website
includes important information on genetic testing and the clinical
trial process. 
About Exon Skipping
 Exon skipping is a potential therapeutic
approach in DMD designed to produce functional dystrophin, a protein
involved in muscle function that is lacking in patients with the
disorder. Each potential therapy based on this technology is designed
to skip a specific exon, and thereby correct for certain genetic
mutations and restore the gene's ability to make a functional, though
shorter, form of the dystrophin protein. Available data suggest that
at least 65 percent of genetic mutations in DMD could potentially be
addressed by exon skipping. 
Eteplirsen is Sarepta's lead investigational exon-skipping drug
candidate in clinical studies, and is designed to skip exon 51 in the
dystrophin gene. Sarepta plans to initiate a confirmatory clinical
trial of eteplirsen in DMD patients with amenable genotypes in the
first quarter of 2014. Eteplirsen is not yet approved or licensed for
use in any country. 
In addition, Sarepta is conducting early-stage research, intended to
support future clinical studies in patients, on drug candidates
targeting exons 53, 50 and 45. Additional research to target other
exons is also planned. 
About Duchenne Muscular Dystrophy
 DMD is an X-linked rare
degenerative neuromuscular disorder causing severe progressive muscle
loss and premature death. One of the most common fatal genetic
disorders, DMD affects approximately one in every 3,500 boys born
worldwide. A devastating and incurable muscle-wasting disease, DMD is
associated with specific errors in the gene that codes for
dystrophin, a protein that plays a key structural role in muscle
fiber function. Progressive muscle weakness in the lower limbs
spreads to the arms, neck and other areas. Eventually, increasing
difficulty in breathing due to respiratory muscle dysfunction
requires ventilation support, and cardiac dysfunction can lead to
heart failure. The condition is universally fatal, and death usually
occurs before the age of 30. 
About Sarepta Therapeutics
 Sarepta Therapeutics is focused on
developing first-in-class RNA-based therapeutics to improve and save
the lives of people affected by serious and life-threatening rare and
infectious diseases. The Company's diverse pipeline includes its lead
program eteplirsen, for Duchenne muscular dystrophy, as well as
potential treatments for some of the world's most lethal infectious
diseases. Sarepta aims to build a leading, independent biotech
company dedicated to translating its RNA-based science into
transformational therapeutics for patients who face significant unmet
medical needs. For more information, please visit us at 
Forward-Looking Statements and Information 
 This press release
includes forward-looking statements, including statements about the
development and clinical status of Sarepta's product candidates and
the potential benefit of such product candidates to Duchenne Muscular
Dystrophy patients. These forward-looking statements involve risks
and uncertainties, many of which are beyond Sarepta's control. Any
such risks can materially and adversely affect the business, results
of operations and the trading price of Sarepta's common stock. For a
detailed description of risks and uncertainties we face, you're
encouraged to review Sarepta's official corporate documents filed
with the Securities and Exchange Commission including the risks and
uncertainties disclosed in Sarepta's latest report on Form 10-Q. We
do not undertake any obligation to publicly update these
forward-looking statements based on events or circumstances after the
date hereof. 
Sarepta Investor Contact: 
Erin Cox 
Sarepta Media Contact:
Jim Baker
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